Medicine

Evolving ASO therapies coming from progression to application

.Contending interests.R.S., M.S., H.G. and also A.A.R. are actually organizers of the 1M1M project. H.G. and also A.A.R. are board of supervisors participants as well as R.S., M.S. as well as A.A.R. are actually participants of the clinical advisory committee of N1C. A.A.R. discloses work by LUMC, which possesses licenses on exon-skipping modern technology, a number of which has actually been actually licensed to BioMarin and consequently sublicensed to Sarepta. As co-inventor of some of these licenses, A.A.R. was allowed to an allotment of royalties. A.A.R. additionally divulges acting as ad hoc consultant for PTC Rehabs, Sarepta Rehabs, Regenxbio, Dyne Therapies, Lilly, BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos, Sapreme, Italfarmaco and also Astra Zeneca. Over the last 5 years, A.A.R. additionally executed ad hoc getting in touch with for Alpha Anomeric. A.A.R. likewise states membership of the scientific boards of advisers of Eisai, Hybridize Therapies, Silence Rehabs, Sarepta Therapies, Sapreme and Mitorx. Over the last 5 years, A.A.R. was also a clinical advisory board participant for ProQR. Commission for A.A.R. u00e2 s consulting as well as advising activities is actually paid to LUMC. Previously 5 years, LUMC likewise received sound speaker gratuity coming from PTC Therapies, Alnylam Netherlands, Italfarmaco and also Pfizer as well as cashing for arrangement research coming from Sapreme, Eisai, Galapagos, Synaffix as well as Alpha Anomeric. Job backing is obtained coming from Sarepta Rehabs and also Entrada through unrestricted gives. H.G. possesses absolutely nothing to disclose relative to the subject matters covered in this document. Before 5 years, he has actually additionally gotten working as a consultant gratuity coming from UCB. M.S. got consultancy gratuity from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly and also Solaxa in the past 5 years, all irrelevant to today document. R.S. possesses absolutely nothing to disclose in connection with the subject matters dealt with in this composition. She has gotten audio speaker and/or working as a consultant honoraria or even funding contributions from Abbvie, Bial, STADA as well as Everpharma in the past 5 years.